Cystinosis

For years, Jordan Janz’s cystinosis demanded around-the-clock care.

Erin Little with her daughter Olivia, 7, who has cystinosis — a deadly genetic disease that destroys her kidneys.

Its lead program is ELX-02, which is in Phase I clinical trial, which focuses on the treatment of cystic fibrosis and cystinosis patients with diagnosed nonsense mutations.

Now, 15 years later, both mother and daughter were at the Natalie’s Wish Celebration at the Fashion Island Hotel on April 15 that raised $3.5 million for cystinosis research.